Dr. Tak Mak and team developing new class of ‘sharpshooter’ cancer drugs

In mid-2013, two of the world's most acclaimed cancer researchers, Drs. Tak Mak of the Princess Margaret Cancer Centre, and Dennis Slamon of the University of California, Los Angeles, announced a major breakthrough in the decade-long pursuit to develop a new class of "sharpshooter" cancer drugs.

"Dr. Slamon and I are proud to stand shoulder-to-shoulder with our fellow scientists and the donors who believe in our vision and have generously helped to finance our critical work," said Dr. Tak Mak, Director of The Campbell Family Cancer Research Institute at the Princess Margaret Cancer Centre. "Today we are excited to announce that we have submitted an Investigational New Drug (IND) application to the Food and Drug Administration for a new drug we have developed based on our research of a target enzyme, PLK4, which plays a crucial role in the process of cell division, particularly in cancer cells. We have been able to progress this project from our initial scientific observations through the complex stages of drug discovery and development to the point where we will shortly be able to test a drug in patients. It is extremely rare for an academic group to have discovered and advanced a novel "first-in-class" drug candidate to this level, and it would not have been possible without the fundamental support provided by donors."

This new drug, CFI-400945, will enter clinical trials phase in the months ahead. In the lab, it has been shown to effectively inhibit the growth of human breast and ovarian cancers as well as colorectal, glioblastoma, lung, melanoma, pancreatic and prostate cancers. Drs. Mak and Slamon are very confident about their submissions to the FDA and Health Canada and anticipate that, following a successful regulatory review, the research team will be able to move quickly to the clinical testing stage. It should be noted that the team has other compounds in early development directed towards additional novel targets uncovered through basic research that are also in the pipeline.

The key supporters of this important discovery research and the development of CFI-400945 have been the walkers and their donors in the Weekend To End Women's Cancers®, the Campbell family, the Canadian Institutes of Health Research (CIHR), Genome Canada and The California Institute for Regenerative Medicine (CIRM). "The incredible fact that this drug has been developed solely with donor and grant dollars makes it an even more extraordinary accomplishment," noted Dr. Mak, one of the most cited cancer research scientists in the world who garnered international recognition by cloning the T-cell receptor, the Holy Grail of cellular immunology. "I truly believe in this important discovery and its therapeutic potential for cancer patients," stated Dr. Slamon, best known for his discovery and development of the cancer drug Herceptin. "I am also astounded by the philanthropic generosity of Princess Margaret donors who believe in this project just as much as our teams working in the labs here in Toronto at The Princess Margaret and at UCLA. This milestone could never have been achieved without their support and belief in our collaborative team. I have had the pleasure of attending several events for The Shoppers Drug Mart Weekend To End Women's Cancers. While addressing the crowds, I have witnessed the passion of the walker community."

"This truly amazing accomplishment has been led by two prolific innovators in the world of cancer research," stated Paul Alofs, President & CEO of The Princess Margaret Cancer Foundation. "Their commitment and a decade of unwavering focus to uncover the important keys to conquering cancer is outstanding. While there is still risk and a long way to go in this new drug discovery process, what this team has been able to achieve is nothing short of incredible. Let me be clear, without the support of our walkers and their donors, as well as the transformational generosity of the Campbell family, this breakthrough could not have taken place. Our supporters believe passionately in The Princess Margaret's ability to lead the revolution in Personalized Cancer Medicine."

Dr. Robert Bell, President and CEO of UHN, is very encouraged by this significant announcement. "To be a leader and innovator takes courage, brilliance and persistence, and from these attributes, great innovation is born. This type of discovery has the potential to change patient outcomes worldwide. The journey from the bench to the bedside continues at the Princess Margaret Cancer Centre. I can assure walkers, donors and funders that they will continue to see direct impact on the revolution that is occurring in Personalized Cancer Medicine."